Emerging Treatments for Pediatric Leukodystrophies

Pediatric Clinics of North America

Volumn 62, Issue 3, 2015, Pages 649-666

  Helman, Guy ^a   Van Haren, Keith ^b   Escolar, Maria L ^c   Vanderver, Adeline ^a,c  

^a CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^b STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c GEORGE WASHINGTON UNIVERSITY SCHOOL OF MEDICINE AND HEALTH SCIENCES   (United States)

Author keywords

Disease modifying; Gene therapy; Genomics; Leukodystrophy; Stem cell; Symptomatic; Therapy

Indexed keywords

3',4',7 TRIHYDROXYISOFLAVONE; ACETYLCYSTEINE; ALEMTUZUMAB; ALPHA TOCOPHEROL; BUSULFAN; CELECOXIB; CEREBROSIDE SULFATASE; CHENODEOXYCHOLIC ACID; CLOFARABINE; CORTICOSTEROID; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; FILGRASTIM; FLUDARABINE; GRANULOCYTE COLONY STIMULATING FACTOR; HYDROXYUREA; LOBELINE; LORENZO OIL; MELPHALAN; METHYLPREDNISOLONE; MEVINOLIN; MYCOPHENOLATE MOFETIL; N ACETYLASPARTIC ACID; RECOMBINANT GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR; SOBETIROME; THIOCTIC ACID; THYMOCYTE ANTIBODY; TRIACETIN; TRIHEPTANOIN;

ADRENOLEUKODYSTROPHY; ADULT POLYGLUCOSAN BODY DISEASE; AICARDI GOUTIERES SYNDROME; ALEXANDER DISEASE; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; CANAVAN DISEASE; CEREBROTENDINOUS XANTHOMATOSIS; CHILD CARE; CLINICAL FEATURE; DIAGNOSTIC TEST; DISEASE ASSOCIATION; ENZYME REPLACEMENT; FUCOSIDOSIS; GENE SEQUENCE; GLOBOID CELL LEUKODYSTROPHY; HUMAN; KINESIOTHERAPY; LEUKODYSTROPHY; METACHROMATIC LEUKODYSTROPHY; MISSENSE MUTATION; NEWBORN SCREENING; PELIZAEUS MERZBACHER DISEASE; PEROXISOMAL BIOGENESIS DISORDER; POLYMERASE III RELATED LEUKODYSTROPHY; REVIEW; TREATMENT INDICATION; X LINKED ADRENOLEUKODYSTROPHY; ZELLWEGER SYNDROME; CHILD; CLINICAL TRIAL (TOPIC); GENETICS; GENOMICS; GENOTYPE; HEREDITARY CENTRAL NERVOUS SYSTEM DEMYELINATING DISEASES; NEUROIMAGING; PHENOTYPE; QUALITY OF LIFE;

CHILD; CLINICAL TRIALS AS TOPIC; GENOMICS; GENOTYPE; HEREDITARY CENTRAL NERVOUS SYSTEM DEMYELINATING DISEASES; HUMANS; NEUROIMAGING; PHENOTYPE; QUALITY OF LIFE;

EID: 84929965856     PISSN: 00313955     EISSN: 15578240     Source Type: Journal    
DOI: 10.1016/j.pcl.2015.03.006     Document Type: Review

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